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NCT Number: NCT07088991

Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia

Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)

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Key information

Conditions

Neurofibromatosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses

Sex eligibility

All sexes

Study type

Observational

Primary location

Research Site, Arkhangelsk, Russia

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About this study

Open-label single-arm, non-interventional, multi-center, cohort study for evaluation of clinical and patient reported outcomes in routine care settings

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 years at the time of inclusion.
  • Signed and dated written informed consent in accordance with ICH-GCP and local law prior to inclusion in the study.
  • NF1 diagnosed (according to the international consensus criteria for evaluating NF1 [18]), see Appendix A.
  • Confirmed PN by clinical assessment, USI, MRI, biopsy; one method is sufficient (histologic confirmation of PN is not necessary in the presence of radiographic findings).
  • Existing of PN-associated symptoms.
  • Adult patients (≥18 years) with newly diagnosed PN or established PN naïve to MEK-inhibitor therapy verified by medical records/histories (e.g., prior prescriptions, hospitalization data).

Exclusion criteria

  • The participation in any clinical study currently (patients participating in other non-interventional studies may be included);
  • Patients with the evidence of a malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy.
  • In the opinion of the investigator the patient is not able to return for follow-up visits or obtain required follow-up studies.
  • Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial, if they are unable to undergo radiographic evaluations or MRI scans requested for research purposes, or other studies which might negatively impact on the pregnancy.
  • Prior receipt of any MEK-inhibitor for PN therapy within 4 months before screening or initiation of therapy prior to age 18 verified by medical records/histories (e.g., prior prescriptions, hospitalization data).
  • Patients who modify their index pathogenetic therapy regimen during the study (for example, pathogenetic antitumor therapy of PN; switching to a different therapy), as determined by the investigator at any protocol-specified visit during the a study, will be excluded from the primary efficacy analysis population from the point of modification onward and will not continue study participation.

Treatment and study plan

Primary outcomes

  1. Age (full years) at the inclusion into the study (date of Visit 1)

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  2. Age (full years) at the primary NF1 diagnosis

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  3. Proportion of men and women

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  4. Body mass index (BMI), kg/m2;

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  5. Body surface area (BSA), m2

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  6. Proportion of patients with each educational status (higher, secondary special, secondary general);

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  7. Proportion of patients from each residential region (federal district of Russia);

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  8. Other than PN specific NF1 symptoms and manifestations

    Time frame: Day 0 (Visit 1)

    skin symptoms and manifestations, tumor manifestations (except malignancies), neurological and cognitive manifestations, cardiovascular manifestations, endocrine manifestations, orthopedic manifestations (distinctive osseous lesions), ophthalmological manifestations, NF1 mutation, presence of NF1 diagnostic criteria in one of parents) (frequency distribution at baseline)

  9. NF1-associated complications

    Time frame: Day 0 (Visit 1)

    malignancy, hypertension, vasculopathy, bone symptoms, chronic neurological conditions, neuropathy, and pain

  10. Non-specific NF1-comorbidities, coded by MedDRA system organ class (SOC) and preferred term (PT)

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  11. Proportion of patients with various specific and non-specific NF1-comorbidities

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  12. Proportion of patients with various PN-associated symptoms

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  13. Proportion of patients with various ECOG score

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  14. Proportion of patients with various PN locations

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  15. PN volume (established by MRI)

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  16. Duration of PN symptoms persistence before inclusion into the study

    Time frame: Day 0 (Visit 1)

    to be calculated between the date when initially symptoms of PN appeared and the date of Visit 1

  17. Duration of PN diagnosis before inclusion into the study

    Time frame: Day 0 (Visit 1)

    to be calculated between the date of primary diagnosis of PN and the date of Visit 1

  18. Medical history of PN

    Time frame: Day 0 (Visit 1)

    size, symptoms, previous medical interventions, surgeries or treatments

  19. Previous physical, instrumental and laboratory examination results

    Time frame: Day 0 (Visit 1)

    To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.

  20. Amount and duration of hospitalizations associated with NF1 according to the investigator's opinion, collected from the medical history since the date of NF1 diagnosis

    Time frame: Day 0 (Visit 1)

    if applicable, to be calculated in patients with at least one such hospitalization

  21. Degree of disability at baseline

    Time frame: Day 0 (Visit 1)

    if applicable, to be calculated in patients with presence of disability at Visit 1

  22. Changing in disability degree during the study

    Time frame: Day 0 (Visit 1)

    if applicable, to be calculated in patients with presence of disability at Visit 1

Study contacts

Contact information is provided by the study sponsor or research team.

AstraZeneca Clinical Study Information Center

CONTACT

[email protected]

1-877-240-9479

Sponsors and collaborators

Lead sponsor

AstraZeneca

Industry

Registry information

Official study title

Clinical and Demographic Characteristics of Adult Patients With NEurofibromatosis in RUSsia

Acronym: NEREUS

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Jul 28, 2025
Registry last updated
Jul 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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